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Trial record 17 of 32 for:    CYSTEAMINE

Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00001213
Recruitment Status : Completed
First Posted : November 4, 1999
Results First Posted : July 22, 2014
Last Update Posted : July 22, 2014
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystinosis
Intervention Drug: Cysteamine
Enrollment 328
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cysteamine Topical Solution
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Cysteamine topical solution administered hourly while awake in both eyes

Cysteamine

Period Title: Overall Study
Started 328
Completed 310
Not Completed 18
Reason Not Completed
Death             18
Arm/Group Title Cysteamine Topical Solution
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Cysteamine topical solution administered hourly while awake in both eyes

Cysteamine

Overall Number of Baseline Participants 328
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 328 participants
13.1  (10.04)
[1]
Measure Description: Although 328 participants were initially enrolled, the mean and standard deviation were only obtained for 322 participants.
Gender   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 328 participants
Female 153
Male 173
[1]
Measure Description: Gender was captured for 326 out of 328 participants, the gender of two participants is unknown.
1.Primary Outcome
Title Number of Participants With Serious and Non-Serious Adverse Events
Hide Description Since efficacy of ophthalmic cysteamine was established and a New Drug Application (NDA) filed, the post-hoc primary outcome measure is the evaluation of safety information. There was no specified time frame for this outcome measure, as safety data was being collected until the drug became available for commercial purchase in May 2013.
Time Frame Any Time Point up to 27 Years
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cysteamine Topical Solution
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Cysteamine topical solution administered hourly while awake in both eyes

Cysteamine

Overall Number of Participants Analyzed 328
Measure Type: Number
Unit of Measure: participants
257
2.Primary Outcome
Title Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response
Hide Description

Response is defined as a decrease from baseline of at least 1 in Corneal Cystine Crystal Score (CCCS) at any time on study when baseline CCCS is greater than or equal to 1, or CCCS does not increase at least 1 at any time on study when baseline CCCS is less than 1.

The CCCS is based on a library of slit-lamp photographs of corneas with increasing crystal densities (0-3). Slit-lamp photos were to be taken to assess the extent of the corneal crystal accumulation. To minimize bias when assessing the extent of corneal crystal accumulation, photos were centrally graded at the National Eye Institute (NEI) where each photo was graded independently by masked graders. If more than one CCCS was recorded in a given study year, the highest (worst) CCCS value was used for that year.

The results were obtained from a combined analyses of the NIH cysteamine studies evaluating various cysteamine ophthalmic solution formulations from 1986 through 2005.

Time Frame Any Time Point Up to 19 Years
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Hide Analysis Population Description
One hundred sixty-one (161) participants were analyzed in the pre-specified intent-to-treat population [defined as patients who received study medication (between 1986 and 2005), and had a baseline and a post-baseline CCCS value]. After 2005, all participants enrolled received open-label treatment and only safety data was obtained.
Arm/Group Title Cysteamine Topical Solution
Hide Arm/Group Description:

Cysteamine topical solution administered hourly while awake in both eyes

Cysteamine

Overall Number of Participants Analyzed 161
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
321
Measure Type: Number
Unit of Measure: eyes
98
Time Frame Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially.
Adverse Event Reporting Description Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
 
Arm/Group Title Cysteamine Topical Solution
Hide Arm/Group Description

Cysteamine topical solution administered hourly while awake in both eyes

Cysteamine

All-Cause Mortality
Cysteamine Topical Solution
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Cysteamine Topical Solution
Affected / at Risk (%) # Events
Total   11/328 (3.35%)    
Eye disorders   
Optic disc disorder  1  1/328 (0.30%)  1
Blindness  1  1/328 (0.30%)  1
Gastrointestinal disorders   
Intestinal obstruction  1  1/328 (0.30%)  1
Infections and infestations   
Gastrointestinal infection  1  1/328 (0.30%)  1
Nervous system disorders   
Benign intracranial hypertension  1  8/328 (2.44%)  8
Renal and urinary disorders   
Renal failure  1  2/328 (0.61%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cysteamine Topical Solution
Affected / at Risk (%) # Events
Total   257/328 (78.35%)    
Eye disorders   
Photophobia  1  239/328 (72.87%)  239
Conjunctival hyperaemia  1  69/328 (21.04%)  69
Eye pain  1  66/328 (20.12%)  66
Ocular hyperaemia  1  52/328 (15.85%)  52
Eye irritation  1  49/328 (14.94%)  49
Lacrimation increased  1  23/328 (7.01%)  23
Optic disc disorder  1  19/328 (5.79%)  19
Vision blurred  1  20/328 (6.10%)  20
Keratitis  1  19/328 (5.79%)  19
Dry eye  1  14/328 (4.27%)  14
Eye pruritis  1  13/328 (3.96%)  13
Conjunctivitis  1  12/328 (3.66%)  12
Eyelid oedema  1  12/328 (3.66%)  12
Retinal disorder  1  11/328 (3.35%)  11
Blindness  1  9/328 (2.74%)  9
Corneal epithelium disorder  1  6/328 (1.83%)  6
Blepharitis  1  5/328 (1.52%)  5
Erythema of eyelid  1  5/328 (1.52%)  5
Eye swelling  1  3/328 (0.91%)  3
Conjunctival oedema  1  2/328 (0.61%)  2
Eye oedema  1  1/328 (0.30%)  1
Eyelid cyst  1  1/328 (0.30%)  1
Eyelid irritation  1  1/328 (0.30%)  1
Ulcerative keratitis  1  1/328 (0.30%)  1
Drug ineffective  1  1/328 (0.30%)  1
Gastrointestinal disorders   
Vomiting  1  8/328 (2.44%)  8
Abdominal pain  1  1/328 (0.30%)  1
General disorders   
Instillation site irritation  1  33/328 (10.06%)  33
Instillation site pain  1  22/328 (6.71%)  22
Adverse drug reaction  1  7/328 (2.13%)  7
Instillation site erythema  1  5/328 (1.52%)  5
Swelling  1  2/328 (0.61%)  2
Instillation site lacrimation  1  1/328 (0.30%)  1
Instillation site reaction  1  1/328 (0.30%)  1
Immune system disorders   
Drug hypersensitivity  1  2/328 (0.61%)  2
Infections and infestations   
Eye infection  1  6/328 (1.83%)  6
Conjunctivitis ineffective  1  3/328 (0.91%)  3
Hordeolum  1  3/328 (0.91%)  3
Investigations   
Visual evoked potentials abnormal  1  1/328 (0.30%)  1
Nervous system disorders   
Headache  1  94/328 (28.66%)  94
Visual field defect  1  35/328 (10.67%)  35
Benign intracranial hypertension  1  4/328 (1.22%)  4
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
Due to the progressive nature of the drug's development, this protocol reflects the culmination of cysteamine studies performed at NIH. The combined analyses of the data is presented and not the data or analyses of one individual protocol.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Rachel J. Bishop, MD, Principal Investigator, National Eye Institute
Organization: National Institutes of Health
Phone: 301-402-3771
EMail: bishopra@nei.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) )
ClinicalTrials.gov Identifier: NCT00001213     History of Changes
Other Study ID Numbers: 860062
86-EI-0062
First Submitted: November 3, 1999
First Posted: November 4, 1999
Results First Submitted: June 20, 2014
Results First Posted: July 22, 2014
Last Update Posted: July 22, 2014